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BioWorld - Thursday, January 29, 2026
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Biomarkers

WBP4-related spliceosomopathy identified in patients with severe neurodevelopmental delay

June 14, 2023
The WW domain binding protein 4 (WBP4) is part of the early spliceosomal complex, and it has been previously shown to enhance splicing both in vitro and in vivo, as well as to regulate alternative splicing. At the ESHG meeting, researchers from Hebrew University of Jerusalem presented data from a study that aimed to delineate WBP4 in the context of human pathologies.
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Genetic brain disorders, conceptual 3D illustration
Neurology/Psychiatric

Spinocerebellar ataxia variants show vast heterogeneity

June 14, 2023
By Mar de Miguel
The phenotypic variety of spinocerebellar ataxias (SCAs) not caused by CAG repeat expansion (polyglutamine SCA) is greater than expected. A collaboration directed by scientists of the Paris Brain Institute described seven variants of this disorder in 756 individuals, observing that age at onset and progression by gene and variant can occur from childhood to late adulthood with very different forms of the disease.
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Other news to note for June 14, 2023

June 14, 2023
Additional early-stage research and drug discovery news in brief, from: Alzecure.
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Respiratory

Intraperitoneally or orally administered OLT-1177 improves experimental allergic asthma

June 13, 2023
At the recent EAACI meeting, researchers presented preclinical data for the inflammasome inhibitor OLT-1177 (dapansutrile), which is being evaluated by Olatec Therapeutics LLC for the treatment of inflammatory diseases.
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Neurology/Psychiatric

Hepaitech (Beijing) Biopharma patents new FXR and GPBAR1 agonists

June 13, 2023
Hepaitech (Beijing) Biopharma Technology Co. Ltd. has disclosed bile acids acting as farnesoid X receptor (FXR) and/or G-protein coupled bile...
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Cancer

Elgen Therapeutics discovers new MLL1 inhibitors for cancer

June 13, 2023
Elgen Therapeutics Inc. has described histone-lysine N-methyltransferase 2A (KMT2A, MLL1) inhibitors reported to be useful for the treatment of cancer.
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Cancer

Baiyu Pharmaceutical presents new PARP-1 inhibitors for cancer

June 13, 2023
Chengdu Baiyu Pharmaceutical Co Ltd. has divulged poly(ADP-ribose) polymerase 1 (PARP-1; ARTD1) inhibitors with improved blood-brain barrier penetration ability and reported to be useful for the treatment of cancer.
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Cancer

Chia Tai Tianqing Pharmaceutical describes new BTK degradation inducers

June 13, 2023
Chia Tai Tianqing Pharmaceutical Group Co. Ltd. has identified proteolysis targeting chimeras (PROTACs) comprising cereblon (CRBN) ligands coupled to a Bruton tyrosine kinase (BTK) targeting moiety via linker and thus acting as BTK degradation inducers.
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Cancer

Chinese researchers divulge new EGFR mutant inhibitors for cancer

June 13, 2023
Researchers at Jinan University (Guangdong) and Shanghai Institute of Materia Medica of the Chinese Academy of Sciences have synthesized...
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Tumor microenvironment
Cancer

First-in-class CCR8 antagonist IPG-051 displays good preclinical antitumor activity

June 13, 2023
Chemokine (C-C motif) receptor 8 (CCR8), a member of the β chemokine receptor family, contributes to the recruitment and immunosuppressive function of regulatory T (Treg) cells within the tumor microenvironment.
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